In its strategic plan for 2016, the Personalized Medicine Coalition (PMC) defined 3 overarching goals for the personalized medicine community: 1) highlight the issues facing personalized medicine; 2) identify the best strategies to integrate personalized medicine into healthcare; and 3) promote public policies that encourage investment in personalized medicine, including regulatory reform, supportive reimbursement, and increased funding for research.
So, how are we doing? The year is not over, but we can discern certain themes.
First and foremost, President Obama and Vice President Biden have focused unprecedented attention on the power and promise of personalized medicine. The Precision Medicine Initiative launched in 2015 and the Cancer Moonshot Task Force organized in the spring of this year have galvanized the research community to uncover and potentially share data that might reveal the secrets of individual variation so that more effective therapies may be developed to, in the president’s words, “give us the tools to better understand, prevent and treat everyone’s health needs.”
But these initiatives, as important and as promising as they are, do not address outdated regulatory and reimbursement policies that continue to slow progress. The Centers for Medicare & Medicaid Services (CMS), for example, continues to place downward pressure on personalized medicine diagnostics. CMS’ recently proposed preliminary gapfill payment determinations, for example, could chill new investment in diagnostics and limit patient access to the kinds of treatments the president envisions.
The ongoing value debate also has implications for the field. Personalized medicine is not even an afterthought when it comes to developing the value assessment tools CMS proposes to use to evaluate what pharmaceutical companies may charge for their products. If that does not change, one-size-fits-all conceptions can and will limit access to new treatments for selected subpopulations that can clearly benefit from them. Recognizing a danger to both innovation and patients in these emerging health technology assessment tools, PMC is working on a white paper to remind policymakers, to paraphrase the president, that just because we can understand individual variation does not mean we will treat it accordingly.
Nevertheless, despite skepticism about personalized medicine in some quarters, progress continues. Last year, for example, 28% of all drugs approved by the FDA had biomarker information in their labels, and the failure of Opdivo (nivolumab) as a first-line therapy for advanced lung cancer has revived interest in the business model for personalized medicine.
We know, however, that patients, providers, and payers lag in their knowledge of the developments in personalized medicine. According to a PMC-commissioned survey published in 2014, only one-third of patients are familiar with the principles of personalized medicine, and only 11% have discussed it with their doctors. As such, developing strategies to integrate personalized medicine into healthcare has become one of the highest priorities for the field.
In brief, in both education and advocacy, we have our work cut out for us.